重组腺相关病毒载体(rAAV)基因药物已经开展六十余项(67)临床研究,其安全、高效、稳定、表达持久等特点越来越受到业界的重视,最近的临床试验发现其在治疗先天性黑内障临床研究中呈现出显著疗效更是极大地振奋了人们的信心。临床研究案例的增加使人们对rAAV基因药物有了更为全面、深入的认识。与此同时,也对基因药物提出了更多挑战与要求,尤其是免疫原性和安全性等方面。 More than 60 (67) clinical trials of recombinant adeno-associated virus (rAAV) gene drugs have attracted more and more attention in the industry because of their safety, high efficiency, stability and long-lasting expression. Recent clinical trials have found that they are innate Clinical studies of sexual cataracts showed a significant effect is greatly stimulated people’s confidence. The increase of clinical research cases has led to a more comprehensive and in-depth understanding of rAAV gene drugs. At the same time, it also poses more challenges and requirements for gene drugs, especially in terms of immunogenicity and safety.